EP4021945A4 - Combinatorial adenine and cytosine dna base editors - Google Patents

Combinatorial adenine and cytosine dna base editors Download PDF

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Publication number
EP4021945A4
EP4021945A4 EP20857058.0A EP20857058A EP4021945A4 EP 4021945 A4 EP4021945 A4 EP 4021945A4 EP 20857058 A EP20857058 A EP 20857058A EP 4021945 A4 EP4021945 A4 EP 4021945A4
Authority
EP
European Patent Office
Prior art keywords
combinatorial
adenine
dna base
base editors
cytosine dna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP20857058.0A
Other languages
German (de)
French (fr)
Other versions
EP4021945A2 (en
Inventor
J. Keith Joung
Ronghao ZHOU
Julian GRUNEWALD
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
General Hospital Corp
Original Assignee
General Hospital Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by General Hospital Corp filed Critical General Hospital Corp
Publication of EP4021945A2 publication Critical patent/EP4021945A2/en
Publication of EP4021945A4 publication Critical patent/EP4021945A4/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Enzymes And Modification Thereof (AREA)
EP20857058.0A 2019-08-30 2020-08-31 Combinatorial adenine and cytosine dna base editors Pending EP4021945A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962894612P 2019-08-30 2019-08-30
US202063023192P 2020-05-11 2020-05-11
PCT/US2020/048825 WO2021042062A2 (en) 2019-08-30 2020-08-31 Combinatorial adenine and cytosine dna base editors

Publications (2)

Publication Number Publication Date
EP4021945A2 EP4021945A2 (en) 2022-07-06
EP4021945A4 true EP4021945A4 (en) 2023-11-15

Family

ID=74683678

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20857058.0A Pending EP4021945A4 (en) 2019-08-30 2020-08-31 Combinatorial adenine and cytosine dna base editors

Country Status (3)

Country Link
US (1) US20220290121A1 (en)
EP (1) EP4021945A4 (en)
WO (1) WO2021042062A2 (en)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
CN111093714A (en) 2017-05-25 2020-05-01 通用医疗公司 Deamination using a split deaminase to restrict unwanted off-target base editors
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
EP3921417A4 (en) 2019-02-04 2022-11-09 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing
WO2020191246A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2021158921A2 (en) * 2020-02-05 2021-08-12 The Broad Institute, Inc. Adenine base editors and uses thereof
GB2614813A (en) 2020-05-08 2023-07-19 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
KR20240019759A (en) * 2021-04-21 2024-02-14 아시모브 인코포레이티드 Stable production system for adeno-associated virus production
CN114438110B (en) * 2022-01-25 2023-08-04 浙江大学杭州国际科创中心 Adenine base editor without PAM limitation accurately and construction method thereof
CN114686456B (en) * 2022-05-10 2023-02-17 中山大学 Base editing system based on bimolecular deaminase complementation and application thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL264565B1 (en) * 2016-08-03 2024-03-01 Harvard College Adenosine nucleobase editors and uses thereof
EP3601562A1 (en) * 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
BR112019021719A2 (en) * 2017-04-21 2020-06-16 The General Hospital Corporation CPF1 VARIANT (CAS12A) WITH CHANGED PAM SPECIFICITY
WO2019040650A1 (en) * 2017-08-23 2019-02-28 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
BR112021001904A2 (en) * 2018-08-03 2021-05-04 Beam Therapeutics Inc. multi-effect nucleobase editors and methods of using them to modify a target nucleic acid sequence
EP3921417A4 (en) * 2019-02-04 2022-11-09 The General Hospital Corporation Adenine dna base editor variants with reduced off-target rna editing

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RINA C. SAKATA ET AL: "A single CRISPR base editor to induce simultaneous C-to-T and A-to-G mutations", BIORXIV, 8 August 2019 (2019-08-08), pages 1 - 17, XP055768028, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.1101/729269v1.full.pdf> [retrieved on 20210122], DOI: 10.1101/729269 *

Also Published As

Publication number Publication date
WO2021042062A2 (en) 2021-03-04
US20220290121A1 (en) 2022-09-15
WO2021042062A3 (en) 2021-04-08
EP4021945A2 (en) 2022-07-06

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